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Expert Interview

Slingshot members are talking to an expert! The topic is:

Discussing Regenxbio’s Phase I/II/III CAMPSIITE trial of RGX-121 recent data in Mucopolysaccharidosis Type II Hunter Syndrome

Ticker(s): RGNX

Who's the expert?

A Geneticist with experience in treating Hunter Syndrome.

Interview Questions
Q1.

Please describe your clinical trial practice,how many patients with Type II Hunter Syndrome have you treated, and can you tell us the details about Mucopolysaccharidosis Type II disorder?

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Q2.

Could you please comment on the CSF GAGs key biomarkers of I2S enzyme activity reductions shown in the results?

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Q3.

Could you talk with us about the standard of care in this area, like Enzyme replacement, gene editing or Bone-marrow and stem-cell transplantation?Which treatments are most promising and why?

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Q4.

Can you please discuss the data that supported FDA approval of Pemazyre, f.ex. The CR rate of 78 and 104 days median time to response, how does it compare to standard of care? (median reduction of CSF HS from baseline at week 48: 33.5% in Cohort 1, 52.9% in Cohort 2 and 62.5% in Cohort 3.)

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Q5.

The company touts that a majority of patients demonstrated increases in I2S protein concentration levels in plasma, and total urine GAG measures demonstrated evidence of a systemic effect of RGX-121, independent of ERT treatment. How convincing is this argument to you?

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Q6.

How likely are you to use RGX-121 for your patients? What results would convince you in the future?

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